Rimegepant for Pediatric Migraine

Recruiting at 425 trial locations

Overseen By Pfizer Pfizer CT.gov Call Center

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Pfizer

Must be taking: Prophylactic migraine

Must not be taking: CGRP antagonists

Disqualifiers: Cluster headache, Uncontrolled psychiatric, Substance abuse, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 2 to 48 hours post-dose

2100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of Rimegepant, a migraine treatment, for moderate or severe migraines in children and teenagers. Participants will receive either the actual medication or a placebo (a pill with no active ingredients) to compare results. Children who have experienced migraines for more than six months, with one to eight moderate or severe attacks per month, may be suitable candidates. Participants should distinguish between migraines and other headaches and weigh over 40 kg. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new migraine treatment for children and teenagers.

Will I have to stop taking my current medications?

The trial allows participants to stay on one stable prophylactic migraine medication, as long as it is not a CGRP antagonist. If you are taking a CGRP antagonist, you will need to stop before joining the study.

Is there any evidence suggesting that Rimegepant/BHV3000 is likely to be safe for humans?

Research has shown that rimegepant is generally safe for both adults and children in treating migraines. One study found that a single dose was safe for children aged 6 to 12 years. Another study confirmed that a 75 mg dose effectively relieves migraines and is safe. Side effects are uncommon, making the treatment usually safe to use.

Testing rimegepant in children indicates it has already passed earlier safety checks, providing confidence in its safety for wider testing. While all medicines can have some side effects, rimegepant appears to have a good safety record so far. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for migraine?

Rimegepant is unique because it offers a new approach to treating pediatric migraines by targeting the calcitonin gene-related peptide (CGRP) pathway, which plays a crucial role in migraine development. Unlike traditional treatments such as triptans or over-the-counter pain relievers, rimegepant is administered as an orally disintegrating tablet (ODT), making it easy for children to take without the need for water. Researchers are excited about rimegepant because it not only provides quick relief but also has the potential for fewer side effects compared to some of the more conventional migraine medications.

What evidence suggests that Rimegepant might be an effective treatment for pediatric migraine?

Research has shown that rimegepant, which participants in this trial may receive, effectively treats migraines. In earlier studies, rimegepant provided pain relief within two hours, helping migraine patients feel better. Patients taking rimegepant were more likely to be pain-free compared to those on a placebo. It also reduced the number of migraine days each month with few side effects. This treatment has proven effective and safe for quick migraine relief in everyday situations. ^{6 7 8 9 10}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

This trial is for children and teens aged 6 to under 18 with a history of migraines, weighing over 40 kg. They must be able to tell the difference between migraine and other headaches, may use stable doses of certain migraine prevention meds but not CGRP antagonists, and should have had 1-8 significant migraine attacks per month in the past two months.

Inclusion Criteria

I am between 6 and 17 years old and will not turn 18 during the study.

I have had migraines for more than 6 months.

I've had 1-8 severe attacks a month in the last 2 months, lasting over 3 hours.

Exclusion Criteria

I have had a stable mental health condition for at least 6 months.

I have had or will have surgery with general anesthesia less than 8 weeks before the screening.

I have had surgery on my stomach or intestines that affects how my body processes food.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BHV-3000 or placebo for the acute treatment of moderate or severe migraine

2 to 48 hours post-dose

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Matching placebo
• Rimegepant/BHV3000

Trial Overview The study is testing BHV-3000 (Rimegepant) against a placebo for quickly treating moderate or severe migraines in young people. Participants will randomly receive either the actual medication or a dummy pill without knowing which one they are taking.

How Is the Trial Designed?

Group I: BHV3000 (active drug) Active Control 1 Intervention

BHV3000 (rimegepant) 75 mg or 50 mg ODT

Group II: Placebo Placebo Group 1 Intervention

Matching 75 mg or 50 mg ODT placebo

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Biohaven Pharmaceutical Holding Company Ltd.

Lead Sponsor

Trials

9

Recruited

6,300+

Published Research Related to This Trial

Rizatriptan 5 mg did not show significant effectiveness over placebo for treating a single migraine attack in adolescents, with similar pain relief rates (68.2% for rizatriptan vs. 68.8% for placebo).

However, in a longer-term study, rizatriptan 5 mg was more effective than standard care for treating multiple migraine attacks, achieving pain relief in 77% of cases compared to 64% with standard care, and it was well tolerated throughout both short- and long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rizatriptan 5 mg for the acute treatment of migraine in adolescents: results from a double-blind, single-attack study and two open-label, multiple-attack studies. Visser, WH., Winner, P., Strohmaier, K., et al.[2012]

In a study involving 674 pediatric patients aged 12-17, rizatriptan was found to be generally safe and well tolerated for the long-term acute treatment of migraines, with 66% experiencing any adverse event but only a small percentage discontinuing due to these events.

The efficacy of rizatriptan was consistent over time, with an average of 46.3% of patients achieving pain freedom within 2 hours after taking the medication, indicating its effectiveness in managing migraine attacks in this age group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term open-label safety study of rizatriptan acute treatment in pediatric migraineurs. Hewitt, DJ., Pearlman, E., Hämäläinen, M., et al.[2017]

Migraine in children is diagnosed using similar criteria as in adults, but attacks are often shorter and can be bilateral, highlighting the need for tailored treatment approaches.

Treatment options for pediatric migraines include safe medications like NSAIDs and triptans for acute attacks, as well as preventive medications such as antidepressants and anticonvulsants, alongside biobehavioral therapies for long-term management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pediatric migraine: recognition and treatment. Hershey, AD., Winner, PK.[2022]

In a study involving 13 patients with migraines, rimegepant was found to be well tolerated when used as an acute treatment alongside CGRP monoclonal antibodies (mAbs) for preventive treatment, with no serious adverse events reported.

Out of the patients, 38% experienced mild to moderate adverse events, primarily nasopharyngitis, indicating that while some side effects occurred, they were generally not severe and did not lead to treatment discontinuation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of Rimegepant, an Oral CGRP Receptor Antagonist, Plus CGRP Monoclonal Antibodies for Migraine. Berman, G., Croop, R., Kudrow, D., et al.[2021]

Rimegepant, an oral CGRP receptor antagonist, has shown significant efficacy in treating migraines, providing pain relief and symptom improvement for up to 48 hours after a single dose, based on completed phase II and III trials.

The medication was well tolerated with only mild to moderate adverse events reported, leading to its FDA approval for acute migraine treatment on February 27, 2020.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rimegepant for the treatment of migraine. Negro, A., Martelletti, P.[2021]

Rimegepant, an orally disintegrating tablet approved by the FDA, has been shown in three phase 3 trials to significantly improve pain relief and reduce bothersome migraine symptoms within 2 hours of administration.

This medication offers a new mechanism of action for treating migraines, making it a suitable option for patients who do not respond well to traditional treatments like triptans or who experience adverse effects from them.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rimegepant Orally Disintegrating Tablet for Acute Migraine Treatment: A Review. DeFalco, AP., Lazim, R., Cope, NE.[2021]

Rimegepant significantly improves outcomes for migraine patients, including freedom from pain and pain relief at 2 hours, with odds ratios indicating strong efficacy compared to placebo.

The treatment shows sustained benefits at 24 hours and does not appear to increase the risk of adverse events, suggesting it is a safe option for migraine management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Rimegepant for Migraine Patients: A Meta-analysis of Randomized Controlled Studies. Yang, C., Zhang, Y.[2023]

In a study involving 36 participants, rimegepant showed a significant increase in systemic exposure in individuals with severe hepatic impairment, with up to a 3.9-fold increase in unbound concentrations compared to healthy controls, indicating a need for caution in this population.

The study found that rimegepant is not recommended for adults with severe hepatic impairment due to the increased risk of adverse effects, as evidenced by a higher concentration of the drug in the bloodstream.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rimegepant 75 mg in Subjects With Hepatic Impairment: Results of a Phase 1, Open-Label, Single-Dose, Parallel-Group Study. Bhardwaj, R., Ivans, A., Stringfellow, J., et al.[2023]

Rimegepant, an oral CGRP receptor antagonist, has shown significant efficacy in reducing monthly migraine days (MMDs) and improving health-related quality of life (HRQoL) compared to placebo, based on a study involving 257 participants with a history of 4-14 MMDs.

When compared to injectable anti-CGRP monoclonal antibodies like galcanezumab and erenumab, rimegepant demonstrated similar effectiveness in reducing MMDs and improving migraine-related disability and quality of life, supporting its use for both acute and preventive migraine treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Matching-adjusted indirect comparisons of oral rimegepant versus placebo, erenumab, and galcanezumab examining monthly migraine days and health-related quality of life in the treatment of migraine. Popoff, E., Johnston, K., Croop, R., et al.[2021]

There is a significant need for more pediatric randomized controlled trials for migraine treatment, as current practices are largely based on adult studies and show wide variation among clinicians.

Newly approved treatments for pediatric migraines include over-the-counter anti-inflammatory medications and triptans, which have been shown to be safe and effective for children and adolescents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Newly Approved Agents for the Treatment and Prevention of Pediatric Migraine. Kacperski, J., Hershey, AD.[2022]

Rizatriptan significantly improved pain freedom in children and adolescents aged 12-17 years with migraines, with 30.6% of those treated achieving pain relief compared to 22.0% in the placebo group, indicating its efficacy as an acute treatment option.

The safety profile of rizatriptan was comparable to placebo, with similar rates of adverse events reported within 14 days, suggesting it is generally well tolerated in the pediatric population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and tolerability of rizatriptan in pediatric migraineurs: results from a randomized, double-blind, placebo-controlled trial using a novel adaptive enrichment design. Ho, TW., Pearlman, E., Lewis, D., et al.[2012]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39762740/

Effectiveness and tolerability of rimegepant in the acute ... Our data confirms rimegepant effectiveness and safety in the acute migraine treatment in a real-world setting in a cohort of participants.

2. thejournalofheadacheandpain.biomedcentral.com thejournalofheadacheandpain.biomedcentral.com/articles/10.1186/s10194-024-01935-8

Effectiveness and tolerability of rimegepant in the acute ... Our data confirms rimegepant effectiveness and safety in the acute migraine treatment in a real-world setting in a cohort of participants.

3. headachejournal.onlinelibrary.wiley.com headachejournal.onlinelibrary.wiley.com/doi/full/10.1111/head.14995

Efficacy and safety of rimegepant for the preventive treatment ... Results showed that rimegepant was more effective than placebo in reducing monthly migraine days, with very few adverse effects.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06616194

NCT06616194 | A Study to Learn About the ... The purpose of this study is to learn about the effect of a study medicine called rimegepant in adolescents who have frequent migraine attacks.

5. withpower.com withpower.com/trial/phase-3-migraine-disorders-2021-083f3

Rimegepant for Pediatric Migraine Rimegepant significantly improves outcomes for migraine patients, including freedom from pain and pain relief at 2 hours, with odds ratios indicating strong ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41133671/?utm_source=FeedFetcher&utm_medium=rss&utm_campaign=None&utm_content=10UQVR81oEgDsIjcpRnZHD3nH08KtDs-k9lAnQzLW-xdwTksHV&fc=None&ff=20251101030704&v=2.18.0.post22+67771e2

A phase 1, multicenter, open-label study to evaluate the ... A single weight-adjusted dose of rimegepant ODT demonstrated a favorable safety profile in children aged ≥6 to <12 years with a history of ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03732638

NCT03732638 | Efficacy and Safety Trial of Rimegepant for ... The purpose of this is study is to compare the efficacy of BHV-3000 (rimegepant) to placebo as a preventive treatment for migraine, as measured by the ...

8. pfizerclinicaltrials.com pfizerclinicaltrials.com/nct05156398-pediatric-migraine-trial

Pediatric Migraine clinical trial NCT05156398 | Pfizer This clinical trial will help doctors, scientists, & migraine specialists learn if the study medicine (called rimegepant) can prevent migraine in children ...

9. headachejournal.onlinelibrary.wiley.com headachejournal.onlinelibrary.wiley.com/doi/10.1111/head.15002

A 52‐week open‐label extension study to evaluate the safety ... Multiple clinical studies have demonstrated that a single 75 mg dose of rimegepant is effective and safe for the acute treatment of migraine, ...

10. mountsinai.org mountsinai.org/clinical-trials/long-term-safety-study-of-rimegepant-in-pediatric-subjects-for-acute-treatment-of-migraine

Long-term Safety Study of Rimegepant in Pediatric ... The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of migraine in children and adolescents (≥ 6 to < 18 years of ...